Basilea Pharmaceutica Ltd. announced the reporting of the updated efficacy and safety results from cohort 1 of the phase 2 study FIDES-01, which evaluated its fibroblast growth factor receptor (FGFR) inhibitor, derazantinib, in patients with FGFR2 fusion-positive advanced or metastatic intrahepatic cholangiocarcinoma (iCCA), a type of bile duct cancer, at the Congress of the European Society for Medical Oncology (ESMO), taking place as a virtual meeting from 16 to 21 September 2021. Patients with advanced iCCA have a poor prognosis. With the current chemotherapy standard-of-care, the median overall survival is less than one year. Cohort 1 of FIDES-01 enrolled 103 iCCA patients with confirmed FGFR2 fusions. Since the reporting of first topline results in early February 2021, more patient follow-up data has been obtained, showing improvements in efficacy outcomes over time. At the cut-off date in early August, for the data presented at the ESMO congress, the disease control rate (DCR) was 75.7%, including 22 patients with a partial response as the best objective response, corresponding to an objective response rate (ORR) of 21.4%. Importantly, the progression-free survival (PFS) further increased to 8.0 months (previously: 7.8 months). The time to progression (TTP) with derazantinib was 8.1 months and thus markedly longer when compared to a TTP of only 4.5 months with the previous anti-cancer treatment the patients had received prior to entering the study. Median overall survival was 15.9 months, with follow-up ongoing. As reported at ESMO, derazantinib had a notably well manageable adverse event profile, with a low incidence of class effects such as nail toxicities, stomatitis, hand-foot syndrome and retinal effects.